Our team is focused on basic and clinical research to better understand BPDCN and improve outcomes. Recent research done at Dana-Farber and collaborating institutions has helped better understand BPDCN and has identified new molecular targets for new therapies for BPDCN. Findings include:
- All BPDCN cells overexpress CD123 (interleukin-3 receptor), a cell-surface protein. The BPDCN Center is one of the leaders of a national clinical trial of the targeted immunotoxin tagraxofusp (also known as Elzonris or SL-401), a novel agent that targets the IL-3 receptor (CD123). This led to approval of tagraxofusp as the first drug specifically approved for BPDCN.
- In the laboratory, we have learned how BPDCN becomes resistant to tagraxofusp. This led to a new clinical trial testing the combination of tagraxofusp with azacitidine, a therapy already used in patients with other types of leukemia.
- By studying the characteristics of patients' BPDCN and the treatment received, we have defined factors associated with survival and identified new chemotherapy and targeted therapies that might be active in the disease.
Featured Clinical Trials for BPDCN
17-056: Phase 1 Study of SL-401 in Combination with Azacitidine and Venetoclax in Relapsed/Refractory Acute Myeloid Leukemia (AML) and in Treatment-Naive Subjects with AML Not Eligible for Standard Induction and in Subjects with Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) or SL-401 in Combination with Azacitidine in Patients with High-Risk Myelodysplastic Syndrome (MDS)